by Sean, Valerie, Brooke, Lexi L., Lexi W.
Ashanti de Silva
Ashanti was the WORLD'S FIRST gene therapy patient. She was only four years old when it happened on September 1990. She was infected with the virus ADA.
The gene therapy treatment started with the extraction of her white blood cells. THese cells were then grown in a culture. After a while the doctors would put the retroviral vector containing ADA in the culture. After white blood cells were able to do their work they were inject back into her body. Next, was the hard part... waiting.
This style of gene therapy were first tested with animals and had been a great success. Ashanti's treatment was also a success. She has no side effects to this day. Immune system works like normal now. Although she had to keep taking small doses of medicine for ADA just in case.
A deficiency that hurts the immune system and causes SCID (severe combined immunodeficiency). People with SCID lack all protection for their immune system from bacteria, fungi, and viruses. The infections are mostly caused by “opportunistic” organisms that help keep a regular healthy immune system illness free. Some infections that people with ADA can get could be severe if not deadly. The symptoms of ADA would include; pneumonia, chronic diarrhea, and widespread skin rashes. Children that are affected with this tend to grow more slowly than a healthy child.
ADA is diagnosed within the first six months of life. Without treatment, babies would be lucky to survive past two years old. In about 10-15 percent of cases onset of immune deficiency is delayed six and twenty-four months of age. It can even be delayed until adulthood. Usually later onset cases are less severe. The most they cause is primarily recurrent upper respiratory and ear infections. Over time though, adults could develop chronic lung damage, malnutrition, and other health related issues.
How common is ADA?
It’s very rare to get. It’s estimated that only 1 in 200,000 to 1,000,000 people get it.
This was the world's first gene therapy and it had been a success. Since it had been a success when would that mean that it will be released into the public? Or will it just be continued on certain people until they think it is safe?
This affects the community because now others with this disease have the opportunity to possibly be cured. It is a big step in genetics like any first gene treatment was. Ashanti stood as a role model for others and they now have a little hope for a better life.